Michael Jaskolka, PhD’s Post

We are very excited to provide updated clinical safety and efficacy data from 28 individuals with sickle cell disease treated with renizgamglogene autogedtemcel (reni-cel) in the Phase 1/2/3 RUBY clinical trial at the American Society of Hematology (ASH) annual congress. Participants were at a median (range) of 9.5 (0.7-25.2) months post-reni-cel infusion, with 11 having >1 year follow-up. Of the 28 participants, 27 were VOE-free post-reni-cel infusion. Early total hemoglobin normalization was observed, with mean total hemoglobin increasing from 9.8 g/dL at baseline to 13.8 g/dL at Month 6 (n=18), as well as rapid and sustained improvements in fetal hemoglobin (HbF) to ≥40% and mean corpuscular HbF concentration per F-cells well above the anti-sickling threshold. Allelic editing levels in both peripheral blood nucleated cells and bone marrow–derived CD34+ cells were high, with mean editing levels of 75.1% (n=8) and 87.8% (n=7) at Month 12, respectively. Markers of hemolysis, including absolute reticulocyte count, indirect bilirubin, lactate dehydrogenase, and haptoglobin, improved or normalized by Month 6 and were generally maintained over time. Finally, meaningful improvements in patient-reported outcome domains for pain, physical function, and social roles and activities were observed. Reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant.   If you have any questions or want more information, please reach out or check the links below!   Poster can be found here - https://lnkd.in/eUSdVYyG   More information on the Ruby Trial can be found here - https://lnkd.in/emSvRPqe   Long-term follow up information can be found here - https://lnkd.in/ebuETUd7   #ASH24 #Biotechnology #Cas12a #CellTherapy #ClinicalTrials #CRISPR #GeneEditing #GeneTherapy #Hematology #SickleCellDisease

Check out Orsini’s latest white paper, exploring the dynamic world of classical hematology. From hemophilia to sickle cell disease, we showcase groundbreaking hematology advancements, uncover trends and hurdles in treatment, and spotlight key payor considerations. Download the white paper now! https://hubs.la/Q02tY9z80 #ClassicalHematology #SpecialtyPharmacy #PatientCare #Hemophilia #SickleCellDisease

Check out Orsini’s latest white paper, exploring the dynamic world of classical hematology. From hemophilia to sickle cell disease, we showcase groundbreaking hematology advancements, uncover trends and hurdles in treatment, and spotlight key payor considerations. Download the white paper now! https://hubs.la/Q02tY91v0 #ClassicalHematology #SpecialtyPharmacy #PatientCare #Hemophilia #SickleCellDisease

Check out Orsini’s latest white paper, exploring the dynamic world of classical hematology. From hemophilia to sickle cell disease, we showcase groundbreaking hematology advancements, uncover trends and hurdles in treatment, and spotlight key payor considerations. Download the white paper now! https://hubs.la/Q02tY9z80 #ClassicalHematology #SpecialtyPharmacy #PatientCare #Hemophilia #SickleCellDisease

Check out Orsini’s latest white paper, exploring the dynamic world of classical hematology. From hemophilia to sickle cell disease, we showcase groundbreaking hematology advancements, uncover trends and hurdles in treatment, and spotlight key payor considerations. Download the white paper now! https://hubs.la/Q02tYb110 #ClassicalHematology #SpecialtyPharmacy #PatientCare #Hemophilia #SickleCellDisease

Another good news from #ASH24 A landmark analysis of the phase III GMMG-HD7 trial assessing the impact of induction regimen and post-induction and post-transplant MRD on PFS. Post-induction and post-transplantation MRD negativity led to better outcomes in both the Isatuximab-RVd and RVd arms, and Isa-RVd was associated with a PFS benefit vs RVd in MRD+ patients. American Society of Hematology International Myeloma Foundation Controversies in Multiple Myeloma (COMy)

Research showcased at this year's 6th European CAR-T Cell Meeting highlighted many of the incredible scientific developments that have occurred over the past year, including... 🔹 Promising rCAR-T outcomes for generalized myasthenia gravis 🔹 CD19 CAR-T success in systemic lupus erythematosus 🔹 Recommendations for cell therapy use in autoimmune diseases The conference, hosted by @ The EBMT and @ European Hematology Association (EHA), showed encouraging results across many disease areas, and highlighted the importance of keeping patient access and safety front of mind. Only then can we ensure that these therapies are not only approvable, but accessible and affordable too. Read the conference summary ➡️ https://lnkd.in/eC3cFRJE #ManufacturingBrighterFutures #CellTherapy #GeneTherapy #PatientAccess #Biotechnology

Controlling both #hematocrit and white blood cell count are important strategies in managing #polycythemiaVera. Published in American Society of Hematology journal Blood. https://bit.ly/4bFMoSH

Do carve out time in your busy schedule to meet Jennell and learn about RBC health biomarkers.

Back in December, Dr. Oussama Abla presented his study of: "Langerhans cell histiocytosis: promises and caveats of targeted therapies in high-risk and CNS disease" at the American Society of Hematology Annual Meeting In the video Deanna sat down with him to review the key takeaways from the study, what the challenges are, and how it can take us forward. View the video here: https://lnkd.in/e8JGdGz9